Discoid lupus erythematosus (DLE) is a subset of cutaneous lupus erythematosus (CLE) that represents 57% of cases of CLE. In the US the estimated prevalence of DLE is approximately 133,000 patients which will enable viDA to seek an Orphan Drug Designation from the FDA and EMEA for VTI-1002. DLE is a chronic dermatological disease that can have a significant effect on quality of life due to severe facial scarring, patches of alopecia and or mutilation of the outer ear.
There is a strong unmet need for a safe and effective topical treatment for DLE. Monotherapy or a combination with anti-malarial agents is proposed. VTI-1002 is the first dermatological agent to specifically and potently target the serine protease Granzyme B (GzmB). GzmB is induced in skin keratinocytes by UV-light and is abundant in skin biopsies of DLE patients co-localizing to the lymphocyte infiltrate in the epidermis and dermis proximal to the dermo-epidermal junction (DEJ) and the adnexal structures (hair follicles). There is considerable evidence to suggest that GzmB activity may be involved in all stages of the disease from initiation and inflammation to the scarring and alopecia that occur with healing of the DLE lesions.
The current market is defined by the use of topical corticosteroids and anti-malarials which are relatively inexpensive but of limited therapeutic value due to lack of performance and potential side effects. There remains a strong unmet clinical need in this severe chronic disease that we believe can be addressed by VTI-1002. The anticipated clinical benefits of VTI-1002 should justify a premium price setting for the final topically applied product. Pricing and reimbursement potential will differ in each geographic market. While some estimates for a novel DLE product in the US range as high as $15,000 to $30,000 USD per patient per year , we are initially assuming an annual price per patient of $1,000 to $3,000 USD. Given this and a DLE population in the US of 133,000, we estimate the US commercial market size could vary between $133 million and $399 million per year.
Receiving an orphan drug designation for DLE in the US and/or EU provides significant benefits including protocol assistance by regulatory authorities, market exclusivity, reduced administration fees, and tax incentives. There are additional benefits that may be realized from having an orphan drug designation such as shortened development timelines, reduced clinical trial size, and opportunities for expansion to other orphan and non-orphan indications.
With no treatments for DLE approved in recent years, the pipeline and new therapeutic options for patients remains thin. However, even with several drugs terminating their trials in DLE, therapies in development for other indications are evaluating DLE as an additional disease target. Examples include apremilast (Otezla®) for psoriatic arthritis and Belimumab (Benlysta®) for SLE, which are running subsequent clinical trials in DLE patients for label expansion. Otezla® was approved in March 2014 and is projected to eventually be a $1B drug based on a broader label following clinical studies in other indications.
viDA is focused on taking our topical formulation containing our lead drug candidate, VTI-1002, through the safety and toxicology studies in order to file a new Investigational New Drug and initiate clinical trials within the next 12 months.